The BioMedReports.com FDA Calendar service includes a database with over 400 entries of (1) pending new drug, biological agent, or medical device new product decisions at the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, and sBLA filings); (2) pending new submissions to the FDA; (3) pending complete response letter (CRL) re-submissions to the FDA; and (4) pending clinical trial results.
On 6/8/09, Dyax Corp. (DYAX) announced today that the FDA accepted the Company’s submission in response to the FDA’s March 2009 Complete Response Letter (CRL), which outlined requirements for approval of DX-88 for the treatment of acute attacks of hereditary angioedema (HAE). In connection with the acceptance, the FDA assigned Dyax’s BLA a new PDUFA action date of 12/1/09, which represents a six-month, Class 2 Review. In the CRL received 3/25/09, the FDA requested submission of a Risk Evaluation and Mitigation Strategy (REMS) and additional information with respect to the chemistry, manufacturing and controls (CMC) section of the BLA. Dyax believes these issues are fully addressed in its reply, which was submitted 6/1/09.
On 10/28/09, DYAX reported that its medical and regulatory teams have continued to respond to inquiries from the FDA as the Agency finalizes its review of the pending BLA for DX-88. The FDA has informed DYAX that certain elements of its proposed REMS to assure safe use, particularly the concept of a closed distribution system, will not be required and the Company believes that an approvable REMS will consist primarily of two components, including a medication guide and a communication plan.
On 5/21/09, Human Genome Sciences (HGSI) announced that it submitted a Biologics License Application (BLA) to the FDA for its human monoclonal antibody drug ABthrax (raxibacumab) for the treatment of inhalation anthrax. Raxibacumab is a first-in-class treatment for anthrax, and is being developed under a contract entered into in 2006 with the Biomedical Advanced Research and Development Authority of the Office of the Assistant Secretary for Preparedness and Response, U.S. Department of Health and Human Services (HHS). On 10/27/09, the Anti-Infective Drugs FDA Advisory Committee voted 16-7, with one abstention, that evidence from animal studies predicted the response in humans. However, the FDA Advisory Panel provided guidance that additional data is needed to show benefit over antibiotics.
On 10/28/09, Aldagen made a preliminary SEC S-1 filing as part of plans for an IPO seeking to raise up to $80.5 million as part of a NASDAQ stock listing under ticker ALDH. Aldagen is a regenerative medicine company which is developing adult stem cell therapies that express high levels of the enzyme aldehyde dehydrogenase (ALDH) which has demonstrated the ability to promote the regeneration of a variety of cells in previous studies.
Aldagen is developing ALD-101 to improve engraftment following umbilical cord blood transplants. In March 2008, Aldagen began a pivotal Phase 3 clinical trial of ALD-101 to evaluate its efficacy in improving engraftment following cord blood transplants used to treat a group of progressive, degenerative and often fatal inherited metabolic diseases in children. These diseases develop because blood-forming stem cells in the patient’s bone marrow do not make a particular enzyme. ALD-101 is the population of ALDHbr stem cells that the Company produces from a portion of a cord blood unit using its proprietary technology.
ALD-101 is infused into the patient shortly after the transplant of the remaining portion of the cord blood unit. In a 24-patient Phase 1 clinical trial, Aldagen observed a reduction in the time to platelet and neutrophil engraftment in patients receiving ALD-101 following their cord blood transplant, as compared to similar patients who had received a cord blood transplant without ALD-101 in an earlier independent clinical trial. Aldagen’s pivotal Phase 3 clinical trial of ALD-101 is designed to further evaluate its ability to accelerate engraftment following cord blood transplants in pediatric patients with inherited metabolic diseases. Aldagen expects to complete enrollment of this trial during 1Q11 and to receive the efficacy results of the trial during 2Q11.
Aldagen is developing ALD-301 to treat critical limb ischemia. In 2008, Aldagen completed a 21-patient Phase 1/2 clinical trial of ALD-301 to assess its safety and potential efficacy as a treatment for advanced critical limb ischemia in patients with no treatment options for restoring blood supply to the affected limb, or revascularization. Critical limb ischemia is a condition characterized by significant impairment of blood flow to the legs and feet caused by a blockage of the arteries. Patients with severe cases of critical limb ischemia may experience persistent pain in their lower extremities and may also suffer from severe tissue damage in the affected area.
There are no drugs currently approved by the FDA for the treatment of this condition. For advanced critical limb ischemia patients with no other therapeutic options for improving blood flow, amputation of the affected limb is often the only available clinical option. ALD-301 is the population of ALDHbr stem cells produced using the Company’s proprietary technology to sort a specified quantity of bone marrow collected from the patient receiving the therapy. In August 2009, Aldagen received an SPA concurrence letter from the FDA for the design of a pivotal Phase 3 clinical trial of ALD-301 for the treatment of critical limb ischemia and the Company plans to commence enrollment of the pivotal trial during 2010.
Aldagen is developing ALD-201 to treat ischemic heart failure. Investigators have completed a 20-patient Phase 1 clinical trial of ALD-201 to assess its safety and potential efficacy as a treatment for ischemic heart failure. ALD-201 is the population of ALDHbr stem cells produced using the Company’s proprietary technology to sort a specified quantity of bone marrow collected from the patient receiving the therapy. ALD-201 is injected directly into the patient’s heart muscle. This procedure is currently performed using a specialized catheter. Aldagen is also developing ALD-151 to improve engraftment following cord blood transplants used to treat leukemia and is currently enrolling patients in a Phase 1 clinical trial for this compound.
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